In 2010, Dr. Nicolas Wein received his PhD in molecular biology in the laboratory of Nicolas Levy, MD, PhD, at the Marseille Medical School in France, where he worked on the development of new diagnosis tools for neuromuscular diseases, the discovery of the Minidysferlin and its use for gene transfer, and investigation of the use of exon-skipping for dysferlinopathies. In 2011, he joined the team of Kevin Flanigan, MD, principal investigator in The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, as a postdoctoral scientist. Dr. Wein worked on the identification of a novel IRES in the dystrophin gene and on an out-of-frame exon-skipping approach that can benefit patients with 5’ mutations in the DMD gene. He, Dr. Flanigan and Dr. Waldrop initiated a clinical trial for this subpopulation of patient in early 2020. In 2016 he opened his lab and continued working on neuromuscular disorders such as Duchenne Muscular dystrophy and neurological disorders. His lab has built on his expertise in translational research. His lab continues to integrate basic science and gene therapy skills in order to advance understanding and treatment of the neurological and muscular disorders. His team long-term projects focus on RNA and DNA editing and gene transfer as therapies using adeno-associated virus and U7snRNA, a small nuclear RNA (RNA editing tool) as well as the CRISPR/Cas9 (DNA editing tool).